Patients with lower-risk myelodysplastic syndromes and anemia can derive long-term benefits from erythropoietin and myeloid growth factor hormones, according to a study in Blood.

The data bolster results from earlier studies that also have demonstrated the efficacy of adding myeloid growth factor hormones (granulocyte colony-stimulating factor, G-CSF) to erythropoietin (EPO) and higher EPO doses for enhancing erythroid responses in myelodysplastic syndromes (MDS), said the study's lead author.

“These findings support the use of this cytokine combination [EPO and G-CSF] to help optimize erythroid responses for MDS patients,” said Peter L. Greenberg, MD, a professor of medicine emeritus at Stanford Cancer Center in Stanford, Calif. “We believe long-term data from our prospective randomized study, with a median follow-up of 5.8 years, provide valuable new information.”

Previous reports in patients without MDS have suggested that treatment with EPO and other hematopoietic cytokines is associated with enhanced adverse events. The results of the present trial suggest that patients with MDS may have different biologic and clinical features that are amenable to this approach, he said.

The prospective, randomized phase III study was designed to evaluate the efficacy and long-term safety of EPO with or without G-CSF plus supportive care (53 patients) vs supportive care alone (57 patients). Patients were assigned to several steps and then crossed over on the basis of their response to treatment (see Table). Erythroid responders were supposed to remain on the treatment regimen for one year (Blood 114:2393-2400, 2009).